Overcoming medulloblastoma resistance in kids using in vivo perturb-seq and preclinical therapies
Official title: Overcoming medulloblastoma resistance to treatment using in vivo perturb-seq and preclinical therapeutics
Funded at University of California, San Francisco · NIH-11428652 · Data updated 2026-11-12
A patient-focused study aiming to find new, less toxic ways to treat pediatric medulloblastoma by using advanced genetic screening to identify effective drug targets in preclinical models.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project start | August 1, 2026 |
| Project end | July 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $680,600 |
| NIH project number | 1R01CA316038-01 |
| Project ID | NIH-11428652 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children diagnosed with medulloblastoma or scientists seeking translational advances that could benefit pediatric patients in the future.
✕Less likely to help
Patients with medulloblastoma who do not respond to targeted approaches or who do not have access to experimental therapies may not experience direct benefit.
What this research studies
This project uses a cutting-edge genetic screening method called perturb-seq to understand how different genetic changes affect medulloblastoma cells. By testing potential therapies in preclinical models, the researchers hope to identify treatment options that work against tumors while reducing side effects. The work is grounded in pediatric brain tumor biology and seeks to translate findings into safer, more effective therapies for children affected by this disease. If successful, it could lead to targeted treatments that improve survival and quality of life for patients and survivors.
Why it matters
Potential benefit: If the approach succeeds, children with medulloblastoma could receive therapies that are more effective and less toxic, potentially improving survival and long-term health.
How similar studies have gone: Perturb-seq and related functional-genomics methods have shown promise in linking genetic changes to drug responses, though application to pediatric medulloblastoma is still advancing.
Where this research is happening
-
University of California, San Francisco
San Francisco, United States Active
Researchers
- Principal investigator: Raleigh, David R — University of California, San Francisco
- Study coordinator: Raleigh, David R
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.